Expanded Access Explained

FDA refers to compassionate use as “expanded access.” Form 3926 is titled Individual Patient Expanded Access IND Application. The vast majority of compassionate use applications to FDA fall into this category. If you have just one patient who needs an investigational drug and you are applying for a drug that you have not administered before, you will be applying for a “single patient IND (Investigational New Drug),” also called “single patient access.” FDA uses the terms “expanded access,” “access,” and “treatment use” interchangeably to refer to the use of an experimental drug.

For regulatory purposes, clinical trials are initiated by a “sponsor” who takes responsibility for the conduct of the study. A sponsor can be an individual, a commercial entity such as a pharmaceutical company, an organization, or a governmental agency. You will become the “sponsor of the IND treatment application,” or a “sponsor-investigator.”

When submitting an application, state at the top of the correspondence and on a mailing cover that this is a request for an individual patient IND for treatment use, specifying whether it is an emergency IND or individual patient IND.

Unless FDA notifies you that treatment may begin earlier, there is a 30-day waiting period before treatment with the drug may begin.

While generally individual patient access is limited to a single course of therapy for a specified time, FDA may authorize multiple courses of therapy or chronic therapy for individual patient access if a condition requires extended treatment.

Individual patient expanded access protocol. If a company (IND sponsor) is concerned that the data from the single patient may reflect negatively on an on-going clinical trial, you may ask it to submit a new protocol or treatment plan under an existing IND. This is called a “single patient protocol.”

Alternatively, you can apply for a single patient protocol if you are already a sponsor of an IND and want to add a patient to the existing IND – for instance, you want to treat a second patient with the same investigational drug for which you already have an IND.

Only the sponsor (a company or individual who holds an IND) may apply for a single patient protocol submission. If the company is the sponsor of the existing IND, it can – but rarely does — submit an individual patient access protocol to its existing IND to allow your patient access to the drug. It is also called “protocol amendment” or “access protocol.”

The application process is largely the same as for a single patient IND. Make sure to include a reference to the existing IND in the cover letter. A regulatory project manager at the FDA Review Division responsible for the existing IND can answer your questions.

There is no 30-day waiting period before treatment with the investigational product may begin, but the protocol must be received by FDA and have approval by an IRB before treatment may begin.

Both Single Patient IND and Single Patient Protocol submissions can be emergency or non-emergency.